Clinical Trials

Over the past 40 years, breast cancer treatment has greatly improved due to lessons learned through clinical trials.

Clinical trials test the safety and benefits of new treatments as well as new combinations (or new doses) of standard treatments. They can also study other parts of care including risk reduction, diagnosis and screening.

People volunteer to take part in these research studies. Those who join clinical trials help further the knowledge base that helps improve breast cancer care.

The importance of clinical trials

Whether a new therapy or test becomes part of standard treatment for breast cancer depends largely on clinical trial results.

Clinical trials take place across the country (and around the world) in many types of medical centers and hospitals.

Often, trials are funded by a single agency like the National Cancer Institute (a government agency) and are done at the same time in many sites across the country. These are called cooperative group clinical trials, and allow researchers to increase the number of people in a given study.

Dedicated physicians, researchers and other health professionals, as well as hospitals, medical research centers and funders are all key to clinical trials. However, most important are the participants.

Phases of cancer treatment trials

There are 4 main phases of clinical trials for new breast cancer treatments:

Phase 1 (phase I) trials study whether a new treatment is safe to use over a range of doses.

The treatment may be given to people with different types of cancers.

A phase 1 trial is mainly a drug safety study.

Phase 2 (phase II) trials study how well a treatment works for a certain cancer, such as breast cancer. These trials may include 25-100 people.

After a drug (or other therapy) is found to be effective in treating breast cancer in a phase 2 trial, a phase 3 trial will study it further.

Phase 3 (phase III) trials study how well a new treatment (including surgical procedures) works compared to the standard treatment (standard of care).

Phase 3 trials study the best way to give the new treatment to get the most benefit and whether it’s better than the current standard treatment.

They may look at different doses of the same drug, different drug combinations or different sequences of giving drugs (for example, studying which drug is best to give first).

In phase 3 trials, people are randomized (chosen by chance) to get either the new treatment or the standard treatment. They don't get to choose which treatment they will get. This ensures the study results reflect the true benefits and risks of the new treatment.

Phase 4 (phase IV) trials study the long-term side effects of treatments or answer new questions about the treatment.

Not all clinical trials fall neatly into 1 category. Some trials may be a combination of 2 categories, such as a phase 1/2 or phase 2/3 trial.

Phase 0 (early phase 1) trials

Phase 0 (also called early phase 1) clinical trials are different from other phases of clinical trials because they have no treatment goals and they are not part of the FDA approval process [213].

Phase 0 trials can give information on whether a drug does what it’s expected to do (based on cell and/or animal data) [213-214]. For example, the trial may look at whether the drug can reach the cancer and how cancer cells in people respond to the drug [213-214].

They study a very small dose of a drug in a small number of people (usually fewer than 15 people) and last less than a week [213-215]. The dose of the drug is so small there is no possible treatment benefit [214-215]. However, this also means the chance of side effects is low [214].

Although not common, phase 0 trials can be an important first step in human studies of a new drug treatment.

Benefits

If you have breast cancer, we encourage you to join a clinical trial. Clinical trials offer the chance to try new treatments and possibly benefit from them (except for phase 0 trials).

Learning a new therapy is better than the standard treatment can also help others. As new therapies are developed, they can open doors to other drugs and procedures that may be even more effective.

Some people worry they will get a placebo instead of an effective treatment in a clinical trial. However, placebos aren’t used in metastatic breast cancer clinical trials and aren’t commonly used in non-metastatic breast cancer trials.

Most often in a breast cancer treatment clinical trial, you will get either the new treatment or the standard treatment. So, even if you don’t get the new drug (or other new therapy), your breast cancer will be treated the same as if you weren’t in the trial. Breast cancer trials never use a placebo instead of standard treatment.

Sometimes in a non-metastatic breast cancer trial, you may get the standard treatment plus a placebo rather than the standard treatment plus the new treatment (being studied). Your health care provider or the clinical research staff can tell you if there’s a placebo (in addition to the standard treatment) in the study.

Possible drawbacks

Eligibility

All clinical trials have specific criteria for joining the study, so you may not be eligible for a trial. Or, there may not be a clinical trial currently recruiting participants that’s right for you.

Side effects

The risks of a new treatment may not be fully understood, so there may be some unexpected side effects.

Testing limits these risks as much as possible. However, all the side effects of a new treatment are often not known until after long-term testing and follow-up.

Financial issues

The cost of a new treatment or test being studied is usually paid for by the clinical trial.

Out-of-pocket costs for most clinical trials are the same as those for standard treatment. However, there may be additional costs if the trial is in a different medical center. For example, you may have extra costs related to travel or parking.

Before enrolling in a clinical trial, talk with your insurance provider and find out exactly which costs are covered (and which are not). This ensures you don’t have any unexpected costs, such as out-of-network fees.

Clinical trials at the Clinical Center of the National Institutes of Health (NIH) in Bethesda, MD are free of charge to those who are eligible to join and willing to get treatment there. For more information on these studies, visit the NIH clinical center website or call 1-800-4CANCER.

Susan G. Komen® partners with CancerCare® to offer the Komen Treatment Assistance Program which bridges the gap for underserved individuals who are actively undergoing breast cancer treatment.

With this program, we aim to help those who are facing financial challenges by providing the following to low-income, underinsured or uninsured women across the country: an assessment by an oncology social worker, limited financial assistance, breast cancer education, psychosocial support and information about local resources.

Funding helps women of any age who have been diagnosed with breast cancer, at any stage of the disease. Financial assistance is granted to women who meet pre-determined eligibility criteria. To learn more about this program and other helpful resources, call the Komen Breast Care Helpline at 1-877 GO KOMEN (1-877-465-6636).

Who can join a clinical trial

All clinical trials have specific criteria for joining the study.

For example, each trial will have a list of medical conditions people must have (or not have) to join the study.

Clinical trials aren’t just for people who are undergoing cancer treatment. There are also clinical trials for people who have finished treatment and for those who have never had breast cancer.

It may be helpful to download and print Susan G. Komen®'s Questions to Ask Your Doctor card on clinical trials and take it with you to your next doctor appointment. There’s plenty of space to write down the answers to these questions, which you can refer back to later.

Who sponsors clinical trials

NCI also sponsors groups that study the same cancer topic, such as the American College of Radiology Imaging Network. These groups include hospitals, universities and physicians who work together to study cancer issues.

The Department of Defense and the Department of Veterans Affairs are other government sources of funding for clinical trials.

Other types of research studies

Clinical trials offer the chance to try new treatments that may be more effective than standard treatments.

Other types of studies, such as cohort and case-control studies, don't offer a possible treatment benefit, but they increase our understanding about breast cancer in many ways. For example, they help us learn about risk factors and survivorship.

Our Breast Care Helpline 1-877 GO KOMEN (1-877-465-6636) provides free, professional support services to anyone with breast cancer questions or concerns, including people diagnosed with breast cancer and their families. Calls are answered by a trained and caring staff member Monday through Friday from 9:00 a.m. to 10:00 p.m. ET and from 6:00 a.m. to 7:00 p.m. PT. You can also email the helpline at helpline@komen.org.

Komen Affiliates offer breast health education as well as fund breast cancer programs through local community organizations. Your local Affiliate may also help you find breast cancer resources in your area. Find your local Affiliate.